JP2021511389A - 脊髄性筋萎縮症を治療する方法 - Google Patents

脊髄性筋萎縮症を治療する方法 Download PDF

Info

Publication number
JP2021511389A
JP2021511389A JP2020561604A JP2020561604A JP2021511389A JP 2021511389 A JP2021511389 A JP 2021511389A JP 2020561604 A JP2020561604 A JP 2020561604A JP 2020561604 A JP2020561604 A JP 2020561604A JP 2021511389 A JP2021511389 A JP 2021511389A
Authority
JP
Japan
Prior art keywords
biological sample
sma
neurofilament
pnf
levels
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2020561604A
Other languages
English (en)
Japanese (ja)
Inventor
ウィルドン フェアウェル,
ウィルドン フェアウェル,
ジョン スタロポリ,
ジョン スタロポリ,
グオリン ジャオ,
グオリン ジャオ,
アレクサンダー マッキャンベル,
アレクサンダー マッキャンベル,
クリストファー コーディー ステビンス,
クリストファー コーディー ステビンス,
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Biogen MA Inc
Original Assignee
Biogen MA Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Biogen MA Inc filed Critical Biogen MA Inc
Publication of JP2021511389A publication Critical patent/JP2021511389A/ja
Priority to JP2023108253A priority Critical patent/JP2023118821A/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • G01N33/6896Neurological disorders, e.g. Alzheimer's disease
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/56Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/575Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of three or more carbon atoms, e.g. cholane, cholestane, ergosterol, sitosterol
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/28Neurological disorders
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/52Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Immunology (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Neurology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Neurosurgery (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pathology (AREA)
  • Cell Biology (AREA)
  • Analytical Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Physics & Mathematics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Food Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
JP2020561604A 2018-01-25 2019-01-25 脊髄性筋萎縮症を治療する方法 Pending JP2021511389A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023108253A JP2023118821A (ja) 2018-01-25 2023-06-30 脊髄性筋萎縮症を治療する方法

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201862622027P 2018-01-25 2018-01-25
US62/622,027 2018-01-25
US201862684507P 2018-06-13 2018-06-13
US62/684,507 2018-06-13
US201862738134P 2018-09-28 2018-09-28
US62/738,134 2018-09-28
PCT/US2019/015185 WO2019147960A1 (en) 2018-01-25 2019-01-25 Methods of treating spinal muscular atrophy

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023108253A Division JP2023118821A (ja) 2018-01-25 2023-06-30 脊髄性筋萎縮症を治療する方法

Publications (1)

Publication Number Publication Date
JP2021511389A true JP2021511389A (ja) 2021-05-06

Family

ID=65529774

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020561604A Pending JP2021511389A (ja) 2018-01-25 2019-01-25 脊髄性筋萎縮症を治療する方法
JP2023108253A Pending JP2023118821A (ja) 2018-01-25 2023-06-30 脊髄性筋萎縮症を治療する方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2023108253A Pending JP2023118821A (ja) 2018-01-25 2023-06-30 脊髄性筋萎縮症を治療する方法

Country Status (13)

Country Link
US (2) US11474113B2 (es)
EP (1) EP3743728A1 (es)
JP (2) JP2021511389A (es)
KR (1) KR20200112906A (es)
CN (1) CN112567246A (es)
AU (1) AU2019211446A1 (es)
BR (1) BR112020015082A2 (es)
CL (1) CL2020001936A1 (es)
IL (1) IL276180A (es)
MA (1) MA51678A (es)
MX (1) MX2020007852A (es)
SG (1) SG11202006677XA (es)
WO (1) WO2019147960A1 (es)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3743728A1 (en) 2018-01-25 2020-12-02 Biogen MA Inc. Methods of treating spinal muscular atrophy
MA62935B1 (fr) 2019-07-19 2023-11-30 Biogen Ma Inc Méthodes de traitement ou de prévention de l'amyotrophie spinale
CA3220428A1 (en) * 2021-06-08 2022-12-15 Alector Llc Methods of use of anti-sortilin antibodies
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100267073A1 (en) * 2007-04-13 2010-10-21 Serge Przedborski Stem cell-based culture system for drug development
JP2012526046A (ja) * 2009-05-02 2012-10-25 ジェンザイム コーポレイション 神経変性障害のための遺伝子治療
US20130280733A1 (en) * 2009-10-06 2013-10-24 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Biomarkers for the Diagnosis of ALS
US20150285822A1 (en) * 2014-04-02 2015-10-08 Wisconsin Alumni Research Foundation Therapeutic and diagnostic methods and compositions for neurodegenerative diseases
US20170363643A1 (en) * 2014-09-12 2017-12-21 Biogen Ma Inc. Compositions and methods for detection of smn protein in a subject and treatment of a subject

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5156840A (en) 1982-03-09 1992-10-20 Cytogen Corporation Amine-containing porphyrin derivatives
US4582789A (en) 1984-03-21 1986-04-15 Cetus Corporation Process for labeling nucleic acids using psoralen derivatives
US4563417A (en) 1984-08-31 1986-01-07 Miles Laboratories, Inc. Nucleic acid hybridization assay employing antibodies to intercalation complexes
US5057313A (en) 1986-02-25 1991-10-15 The Center For Molecular Medicine And Immunology Diagnostic and therapeutic antibody conjugates
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
US5210015A (en) 1990-08-06 1993-05-11 Hoffman-La Roche Inc. Homogeneous assay system using the nuclease activity of a nucleic acid polymerase
EP1231282A3 (en) 1990-12-06 2005-05-18 Affymetrix, Inc. Methods and compositions for identification of polymers
US6027880A (en) 1995-08-02 2000-02-22 Affymetrix, Inc. Arrays of nucleic acid probes and methods of using the same for detecting cystic fibrosis
US6156501A (en) 1993-10-26 2000-12-05 Affymetrix, Inc. Arrays of modified nucleic acid probes and methods of use
WO1997046313A1 (en) 1996-06-07 1997-12-11 Eos Biotechnology, Inc. Immobilised linear oligonucleotide arrays
JP4808365B2 (ja) 2001-03-02 2011-11-02 ユニバーシティ オブ ピッツバーグ オブ ザ コモンウェルス システム オブ ハイヤー エデュケイション Pcr法
EP1379545A2 (de) 2001-04-19 2004-01-14 Gesellschaft für biotechnologische Forschung mbH (GBF) Verfahren zur herstellung stabiler, regenerierbarer antikörper-arrays
US6812341B1 (en) 2001-05-11 2004-11-02 Ambion, Inc. High efficiency mRNA isolation methods and compositions
US20030013208A1 (en) 2001-07-13 2003-01-16 Milagen, Inc. Information enhanced antibody arrays
US7252976B2 (en) 2002-08-28 2007-08-07 Board Of Regents The University Of Texas System Quantitative RT-PCR to AC133 to diagnose cancer and monitor angiogenic activity in a cell sample
EP3470072A1 (en) 2005-06-23 2019-04-17 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing
KR20190037378A (ko) 2009-06-17 2019-04-05 바이오젠 엠에이 인코포레이티드 대상에게서 smn2 스플라이싱을 조정하기 위한 조성물 및 방법
EP2943225A4 (en) 2013-01-09 2016-07-13 Ionis Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR MODULATING SMN2 DISTRIBUTION IN THE BODY OF A PATIENT
US10385341B2 (en) 2014-04-01 2019-08-20 Biogen Ma Inc. Compositions for modulating SOD-1 expression
US20170044538A1 (en) 2014-04-17 2017-02-16 Biogen Ma Inc. Compositions and Methods for Modulation of SMN2 Splicing in a Subject
AU2017223706A1 (en) * 2016-02-23 2018-09-20 Indiana University Research & Technology Corporation Combination therapies for treatment of spinal muscular atrophy
US20210172963A1 (en) * 2017-05-26 2021-06-10 University Of Miami Determining onset of amyotrophic lateral sclerosis
WO2019079240A1 (en) * 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
EP3743728A1 (en) 2018-01-25 2020-12-02 Biogen MA Inc. Methods of treating spinal muscular atrophy
EA201992364A1 (ru) * 2018-04-02 2020-03-23 Кохерус Байосайенсис Инк. АГОНИСТ PPARγ ДЛЯ ЛЕЧЕНИЯ ПРОГРЕССИРУЮЩЕГО НАДЪЯДЕРНОГО ПАРАЛИЧА
WO2020061355A2 (en) 2018-09-20 2020-03-26 Biogen Ma Inc. Serum neurofilament protein for guiding therapeutic intervention in multiple sclerosis
BR112021010660A2 (pt) 2018-12-06 2021-08-24 Biogen Ma Inc. Proteína de neurofilamento para guiar intervenção terapêutica sobre esclerose lateral amiotrófica
TW202035694A (zh) 2018-12-14 2020-10-01 美商百健Ma公司 用於治療及預防肌肉萎縮性脊髓側索硬化症之組合物及方法
MA54949A (fr) 2019-02-12 2021-12-22 Biogen Ma Inc Biomarqueurs de la leucoencéphalopathie multifocale progressive

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100267073A1 (en) * 2007-04-13 2010-10-21 Serge Przedborski Stem cell-based culture system for drug development
JP2012526046A (ja) * 2009-05-02 2012-10-25 ジェンザイム コーポレイション 神経変性障害のための遺伝子治療
US20130280733A1 (en) * 2009-10-06 2013-10-24 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Biomarkers for the Diagnosis of ALS
US20150285822A1 (en) * 2014-04-02 2015-10-08 Wisconsin Alumni Research Foundation Therapeutic and diagnostic methods and compositions for neurodegenerative diseases
US20170363643A1 (en) * 2014-09-12 2017-12-21 Biogen Ma Inc. Compositions and methods for detection of smn protein in a subject and treatment of a subject

Also Published As

Publication number Publication date
IL276180A (en) 2020-09-30
CN112567246A (zh) 2021-03-26
KR20200112906A (ko) 2020-10-05
MA51678A (fr) 2020-12-02
AU2019211446A1 (en) 2020-09-10
MX2020007852A (es) 2021-01-29
WO2019147960A1 (en) 2019-08-01
CL2020001936A1 (es) 2020-12-18
SG11202006677XA (en) 2020-08-28
BR112020015082A2 (pt) 2021-01-05
JP2023118821A (ja) 2023-08-25
US20210041459A1 (en) 2021-02-11
US11474113B2 (en) 2022-10-18
EP3743728A1 (en) 2020-12-02
US20230107651A1 (en) 2023-04-06

Similar Documents

Publication Publication Date Title
US11598776B2 (en) Biomarkers for predicting and assessing responsiveness of thyroid and kidney cancer subjects to lenvatinib compounds
US20230107651A1 (en) Methods of treating spinal muscular atrophy
US20180106817A1 (en) Protein biomarkers and therapeutic targets for renal disorders
JP6803230B2 (ja) アルツハイマー病に関するバイオマーカー及び方法
US20230194536A1 (en) Markers for the diagnosis and treatment of non-alcoholic steatohepatitis (nash) and advance liver fibrosis
US20110189698A1 (en) Protein Biomarkers and Methods for Diagnosing Kawasaki Disease
JP2010510528A (ja) 自己免疫疾患のバイオマーカー
US20120178100A1 (en) Serum Markers Predicting Clinical Response to Anti-TNF Alpha Antibodies in Patients with Psoriatic Arthritis
KR20140108718A (ko) 가와사키 질환용 바이오마커
AU2008247705B2 (en) Methods and compositions for diagnosing osteoarthritis in a feline
WO2021228125A1 (en) Protein markers for assessing alzheimer's disease
CN110988351A (zh) 血管细胞黏附分子在制备抑郁症诊断治疗相关产品的用途
KR102547531B1 (ko) 주요 우울 장애 치료 반응성 평가용 바이오마커
US20220128578A1 (en) Biomarkers of progressive multifocal leukoencephalopathy
CN114438191A (zh) 缺氧诱导因子1α作为标志物在抑郁症复发诊断中的应用
JP7016110B2 (ja) スティル病と敗血症との鑑別用バイオマーカー
US20150376723A1 (en) Prognostic biomarkers for influenza
CA3089120A1 (en) Methods of treating spinal muscular atrophy
WO2019053124A1 (en) PROADRENOMEDULINE AS AN INDICATOR FOR RENAL REPLACEMENT THERAPY IN SERIOUSLY PATIENT PATIENTS
EP3502691A1 (en) Proadrenomedullin as indicator for renal replacement therapy in critically ill patients
US20230400473A1 (en) Methods and compositions for the treatment of crohn's disease
US20200190589A1 (en) Use of markers in the diagnosis and treatment of lupus

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200928

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20220113

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230202

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20230501

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230630

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230920

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20231129

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240220

A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20240516